Claims for Patent: 12,594,268

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Summary for Patent: 12,594,268

Title:	Methods of treating Fabry disease in patients having a mutation in the GLA gene
Abstract:	Provided are methods of treating a patient diagnosed with Fabry disease and methods of enhancing α-galactosidase A in a patient diagnosed with or suspected of having Fabry disease. Certain methods comprise administering to a patient a therapeutically effective dose of a pharmacological chaperone for α-galactosidase A, wherein the patient has a mutation in the nucleic acid sequence encoding α-galactosidase A. Also described are uses of pharmacological chaperones for the treatment of Fabry disease and compositions for use in the treatment of Fabry disease.
Inventor(s):	Elfrida Benjamin
Assignee:	Amicus Therapeutics Inc
Application Number:	US17/269,890
Patent Claims:	1. A method for treatment of Fabry disease in a human patient in need thereof, the method comprising administering to the patient a therapeutically effective dose of migalastat or a salt thereof, wherein the patient has an α-galactosidase A mutation selected from the group consisting of: D33H, G35A, Y88S, T194A, W204G, Y216S, Q250K, and R392T. 2. The method of claim 1, wherein the migalastat or salt thereof is administered to the patient every other day. 3. The method of claim 1, wherein the patient is administered 100 to 150 mg free base equivalent of the migalastat or salt thereof every other day. 4. The method of claim 1, wherein the patient is administered 150 mg of migalastat hydrochloride every other day. 5. The method of claim 1, wherein the patient is male. 6. The method of claim 1, wherein the patient is female. 7. The method of claim 1, wherein the mutation is disclosed in a pharmacological reference table. 8. The method of claim 7, wherein the pharmacological reference table is provided in a product label for a migalastat product approved for the treatment of Fabry disease. 9. The method of claim 1, wherein the mutation is D33H.